Brineura (cerliponase alfa)

POLICY NUMBER

L.5.01.515

Brineura (cerliponase alfa)

Please perform a formulary drug search on your patient’s member ID to ensure the prescription drug is covered under their benefit plan. The medication(s) in this medical policy may not be covered under a specific member’s benefit plan.

DESCRIPTION

Neuronal ceroid lipofuscinosis type 2 (CLN2) disease is a neurodegenerative disease caused by deficiency of the lysosomal enzyme tripeptidyl peptidase-1 (TPP1). Deficiency in TPP1 activity results in the accumulation of lysosomal storage materials normally metabolized by this enzyme in the central nervous system (CNS), leading to progressive decline in motor function.

CLN2 occurs in about two to four of every 100,000 live births in the United States. Signs and symptoms usually begin by age 2-4 and include language delay, seizures, ataxia, muscle twitches, and vision loss. Brineura (cerliponase alfa), a recombinant form of TPP1, is indicated to slow the loss of ambulation in pediatric patients with CLN2 disease and is administered into the cerebrospinal fluid (CSF) by a surgically implanted intraventricular access device.

Quantity Limits

POLICY

Brineura does not require prior authorization. However, all claims will be reviewed for medical necessity based on the medical policy guidelines outlined below. 

Initial Criteria

Brineura (cerliponase alfa) may be considered medically necessary when ALL of the following criteria are met:

1. The individual is at least 37 weeks post-menstrual age (gestational age at birth plus post-natal age) and weighs at least 2.5 kg;

2. The individual has a definitive diagnosis of late infantile onset neuronal ceroid lipofuscinosis type 2 (CLN2) disease confirmed by at least ONE of the following:

   1. Deficiency of the lysosomal enzyme tripeptidyl peptidase-1 (TPP1); OR

   2. Molecular analysis indicating two (2) pathogenic variants/mutations in the TPP1/CLN2 gene on chromosome 11p15

      in trans (i.e. separate parental alleles) in the TPP1/CLN2 gene;

3. The individual has mild to moderate disease documented by a two-domain score of 3 – 6 on motor and language domains of the Hamburg CLN2 Clinical Rating Scale, with a score of at least 1 in each of these two domains (See Policy Guidelines);

4. The prescriber is a specialist, or has consulted with a specialist in the area of the individual's diagnosis (e.g., pediatric neurologist, geneticist);

5. The individual does NOT have any FDA labeled contraindications to therapy with the requested agent; AND

6. The prescribed dosage is within the program quantity limits based on FDA approved labeled dosage.

Length of Approval: 12 months

Renewal Criteria

Brineura (cerliponase alfa) may be approved for continued use when ALL of the following are met:

1. The individual has been previously approved for therapy with the requested agent through BCBSMS review process;

2. The individual continues to meet all initial approval criteria;

3. The individual has positively responded to therapy compared to pretreatment baseline (i.e., stability/lack of decline in motor function/milestones on the Motor domain of the Hamburg CLN2 Clinical Rating Scale [Decline is defined as having an unreversed (sustained) 2-category decline or an unreversed score of 0];

4. The prescriber is a specialist, or has consulted with a specialist in the area of the individual's diagnosis (e.g., pediatric neurologist, geneticist);

5. The individual does not have any FDA-labeled contraindication(s) to therapy with the requested agent; AND

6. The prescribed dosage is within the program quantity limits based on FDA approved labeled dosage.

Length of Approval: 12 months 

Brineura (cerliponase alfa) is considered investigational for all other indications.

POLICY EXCEPTIONS

State Health Plan (State and School Employees) Participants

POLICY GUIDELINES

The coverage guidelines outlined in the Medical Policy Manual should not be used in lieu of the Member's specific benefit plan language.

Medically Necessary is defined as those services, treatments, procedures, equipment, drugs, devices, items or supplies furnished by a covered Provider that are required to identify or treat a Member's illness, injury or Mental Health Disorders, and which Company determines are covered under this Benefit Plan based on the criteria as follows in A through D:

1. consistent with the symptoms or diagnosis and treatment of the Member's condition, illness, or injury; and

2. appropriate with regard to standards of good medical practice; and

3. not solely for the convenience of the Member, his or her Provider; and

4. the most appropriate supply or level of care which can safely be provided to Member. When applied to the care of an Inpatient, it further means that services for the Member's medical symptoms or conditions require that the services cannot be safely provided to the Member as an Outpatient.

For the definition of medical necessity, “standards of good medical practice” means standards that are based on credible scientific evidence published in peer-reviewed medical literature generally recognized by the relevant medical community, and physician specialty society recommendations, and the views of medical practitioners practicing in relevant clinical areas and any other relevant factors. BCBSMS makes no payment for services, treatments, procedures, equipment, drugs, devices, items or supplies which are not documented to be Medically Necessary. The fact that a Physician or other Provider has prescribed, ordered, recommended, or approved a service or supply does not in itself, make it Medically Necessary.

BCBSMS may request medical records for determination of medical necessity. When medical records are requested, letters of support and/or explanation are often useful, but are not sufficient documentation unless all specific information needed to make a medical necessity determination is included.

Investigative is defined as the use of any treatment procedure, facility, equipment, drug, device, or supply not yet recognized as a generally accepted standard of good medical practice for the treatment of the condition being treated and; therefore, is not considered medically necessary. For the definition of Investigative, “generally accepted standards of medical practice” means standards that are based on credible scientific evidence published in peer-reviewed medical literature generally recognized by the relevant medical community, and physician specialty society recommendations, and the views of medical practitioners practicing in relevant clinical areas and any other relevant factors. In order for equipment, devices, drugs or supplies [ie, technologies], to be considered not investigative, the technology must have final approval from the appropriate governmental bodies, and scientific evidence must permit conclusions concerning the effect of the technology on health outcomes, and the technology must improve the net health outcome, and the technology must be as beneficial as any established alternative and the improvement must be attainable outside the testing/investigational setting.

Scoring for Motor and Language Function on the CLN2 Clinical Rating Scale

POLICY HISTORY

01/25/2018: New policy added.

03/27/2018: Policy reviewed and approved by Pharmacy & Therapeutics (P&T) Committee.

12/19/2018: Code Reference section updated to add new HCPCS code J0567, effective 01/01/2019.

07/29/2020: Policy reviewed and approved by Pharmacy & Therapeutics (P&T) Committee. Added drug name to the top of the policy. Policy description updated. Initial approval criteria revised to combine statements regarding diagnosis of late infantile onset neuronal CLN2 TPP1 deficiency. Renewal evaluation criteria revised to state that the patient has positively responded to therapy compared to pretreatment baseline (i.e., a 2-category improvement or an improvement from 0 in the Motor domain of the CLN2 Clinical Rating Scale). Not Medically Necessary statement changed to investigational. Sources updated.

07/13/2021: Policy description updated to add indications for Brineura and to add the generic name to the quantity limits table. Policy section updated to revise the initial and renewal criteria. State Health Plan members removed from Policy Exceptions. Policy Guidelines updated to change "Nervous/Mental Conditions" to "Mental Health Disorders" and "Medically Necessary" to "medical necessity." Added information regarding BCBSMS request for medical records and defined investigative. Sources updated.

07/01/2023: Policy Exceptions updated regarding State Health Plan (State and School Employees) Participants.

10/01/2024: Policy reviewed and approved by the Pharmacy & Therapeutics (P&T) Committee. Policy updated to change "member" to "individual." Policy Guidelines updated to add scoring for motor and language function. Sources updated.

09/09/2025: Policy reviewed and approved by the Pharmacy & Therapeutics (P&T) Committee. Policy description updated regarding neuronal ceroid lipofuscinosis type 2 disease and indications for Brineura (cerliponase alfa). Initial criteria revised to update the following: 1) age and weight requirement; 2) revised criteria to confirm diagnosis of late infantile onset neuronal ceroid lipofuscinosis type 2 (CLN2) disease. Renewal criteria revised regarding the individual's positive response to therapy. Sources updated.

SOURCE(S)

1. Brineura prescribing information. BioMarin Pharmaceutical Inc. August 2024. Last accessed June 2025.

2. Schulz A, Ajayi T, Specchio N, et al. CLN2 Study Group. Study of Intraventricular Cerliponase Alfa for CLN2 Disease. N Engl J Med. 2018 May 17;378(20):1898-1907. doi: 10.1056/NEJMoa1712649. Epub 2018 Apr 24. PMID: 29688815. 

CODE REFERENCE

This may not be a comprehensive list of procedure codes applicable to this policy.

The code(s) listed below are ONLY medically necessary if the procedure is performed according to the "Policy" section of this document.

Medically Necessary Codes

CPT copyright American Medical Association. All rights reserved. CPT is a registered trademark of the American Medical Association.